Acute Hepatic PorphyriaNCT03547297
INSIGHT-AHP: A Study to Characterize the Prevalence of Acute Hepatic Porphyria (AHP) in Patients With Clinical Presentation and History Consistent With AHP
An observational study of Acute Hepatic Porphyria and Hepatic Porphyrias, run by Alnylam Pharmaceuticals at 12 sites in United States.
Trial summary
This study will use specific diagnostic tests on a group of patients who are experiencing symptoms typical of acute hepatic porphyria (AHP) to determine how many have the condition, and to potentially help improve the diagnostic process for patients in the future. Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Acute Hepatic Porphyria, Hepatic Porphyrias, Acute Intermittent Porphyria (AIP)
- How many people
- 212 participants
- Where
- 12 sitesUnited States
- Study dates
- 29 May 2018to 15 Jan 2019
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Male and female participants age ≥ 16 years who present with symptoms consistent with Acute Hepatic Porphyria such as recurrent and severe episodes of abdominal pain.
Who may be able to join
- ≥ 16 years of age
- Recurrent and severe episodes of abdominal pain that last for at least 24 hours
- At least 1 episode of severe abdominal pain that required an urgent healthcare visit in the past 12 months
- Other symptoms related to AHP that your doctor will discuss with you
- Willing and able to comply with protocol required assessments and provide written informed consent
Who cannot join
- Known diagnosis of AHP
- Alternative explanation for abdominal pain with clinical response to treatment specific for alternative diagnosis
Trial locations and contacts
12 sites in 1 country.
12 sites in 1 country
United States12 sites
Clinical Trial Site
Ames, Iowa, 50010
Clinical Trial Site
Houston, Texas, 77025
Clinical Trial Site
Jacksonville, Florida, 32256
Clinical Trial Site
Knoxville, Tennessee, 37912
Clinical Trial Site
Las Vegas, Nevada, 89106
Clinical Trial Site
Manhasset, New York, 11030
Clinical Trial Site
Margate, Florida, 33063
Clinical Trial Site
Pasadena, California, 91101
Clinical Trial Site
Pittsburgh, Pennsylvania, 15232
Clinical Trial Site
Pottsville, Pennsylvania, 17901
Clinical Trial Site
Salt Lake City, Utah, 84124
Clinical Trial Site
Winston-Salem, North Carolina, 27103
About this study
This study will use specific diagnostic tests on a group of patients who are experiencing symptoms typical of acute hepatic porphyria (AHP) to determine how many have the condition, and to potentially help improve the diagnostic process for patients in the future.
- Conditions
- Acute Hepatic Porphyria, Hepatic Porphyrias, Acute Intermittent Porphyria (AIP), Hereditary Coproporphyria (HCP), Variegate Porphyria (VP), ALA Dehydratase Deficient Porphyria (ADP)
- Keywords
- Acute Hepatic Porphyria, Hepatic Porphyrias, Acute Intermittent Porphyria (AIP), Hereditary Coproporphyria (HCP), Variegate Porphyria (VP), ALA Dehydratase Deficient Porphyria (ADP), Porphyria Acute
- Medical subject headings
- Coproporphyria, Hereditary, Porphyrias, Hepatic, Porphyria, Acute Intermittent, Porphyria, Variegate
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of participants with current or past clinically symptomatic AHP with urinary aminolevulinic acid (ALA) or porphobilinogen (PBG) values above the upper limit of normal (ULN)
Measured: 12 months
Secondary outcomes (6)
Proportion of participants with AHPs that contain likely pathogenic mutation(s) in the presence of urinary ALA or PBG values above the ULN
Measured: 12 months
Proportion of patients with pathogenic mutation(s) in the presence of urinary ALA and PBG values above the ULN
Measured: 12 months
Proportion of patients with variants of unknown significance in the presence of urinary ALA and PBG values above the ULN
Measured: 12 months
Proportion of patients with negative genetic tests in the presence of urinary ALA or PBG values above the ULN
Measured: 12 months
Correlation of biochemical and genetic test results with severity of disease measured by symptom frequency and severity in the participant questionnaire
Measured: 12 months
Correlation of biochemical and genetic test results with severity of disease measured by healthcare utilization in the participant questionnaire
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Jun 2018
- Study start29 May 2018
- Primary completion15 Jan 2019
- Study completion15 Jan 2019
Why it stopped: Sponsor elected not to continue with study
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Hepatic PorphyriaStudies of the same condition, open and under way.
- More trials run by Alnylam PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 212 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alnylam Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Quinn Dinh, MD, Study director, Alnylam Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03547297
- Sponsor's protocol ID
- ALN-AS1-NT-002
- Status last verified
- Jan 2019
- First submitted
- 18 May 2018
- Last updated
- 25 Jan 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 25 Jan 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- INSIGHT-AHP: A Study to Characterize the Prevalence of Acute Hepatic Porphyria (AHP) in Patients With Clinical Presentation and History Consistent With AHP
- ClinicalTrials.gov ID
- NCT03547297
- Acronym / sponsor ID
- ALN-AS1-NT-002
- Conditions
- Acute Hepatic Porphyria, Hepatic Porphyrias, Acute Intermittent Porphyria (AIP), Hereditary Coproporphyria (HCP), Variegate Porphyria (VP), ALA Dehydratase Deficient Porphyria (ADP)
- Keywords
- Acute Hepatic Porphyria, Hepatic Porphyrias, Acute Intermittent Porphyria (AIP), Hereditary Coproporphyria (HCP), Variegate Porphyria (VP), ALA Dehydratase Deficient Porphyria (ADP), Porphyria Acute
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Sponsor elected not to continue with study
- Enrolment
- 212 participants (actual)
- Lead sponsor
- Alnylam PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 29 May 2018 (actual)
- Primary completion
- 15 Jan 2019 (actual)
- Study completion
- 15 Jan 2019 (actual)
- First posted
- 6 Jun 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study will use specific diagnostic tests on a group of patients who are experiencing symptoms typical of acute hepatic porphyria (AHP) to determine how many have the condition, and to potentially help improve the diagnostic process for patients in the future.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Male and female participants age ≥ 16 years who present with symptoms consistent with Acute Hepatic Porphyria such as recurrent and severe episodes of abdominal pain.
Key inclusion criteria (5 listed)
- ≥ 16 years of age
- Recurrent and severe episodes of abdominal pain that last for at least 24 hours
- At least 1 episode of severe abdominal pain that required an urgent healthcare visit in the past 12 months
- Other symptoms related to AHP that your doctor will discuss with you
- Willing and able to comply with protocol required assessments and provide written informed consent
Key exclusion criteria (2 listed)
- Known diagnosis of AHP
- Alternative explanation for abdominal pain with clinical response to treatment specific for alternative diagnosis
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Proportion of participants with current or past clinically symptomatic AHP with urinary aminolevulinic acid (ALA) or porphobilinogen (PBG) values above the upper limit of normal (ULN)
Time frame: 12 months
6 secondary outcomes
Proportion of participants with AHPs that contain likely pathogenic mutation(s) in the presence of urinary ALA or PBG values above the ULN
Time frame: 12 months
Proportion of patients with pathogenic mutation(s) in the presence of urinary ALA and PBG values above the ULN
Time frame: 12 months
Proportion of patients with variants of unknown significance in the presence of urinary ALA and PBG values above the ULN
Time frame: 12 months
Proportion of patients with negative genetic tests in the presence of urinary ALA or PBG values above the ULN
Time frame: 12 months
Correlation of biochemical and genetic test results with severity of disease measured by symptom frequency and severity in the participant questionnaire
Time frame: 12 months
Correlation of biochemical and genetic test results with severity of disease measured by healthcare utilization in the participant questionnaire
Time frame: 12 months
Results and safety
The study was stopped early (reason given: Sponsor elected not to continue with study). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Jan 2019.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Quinn Dinh, MD study director
Alnylam Pharmaceuticals
12 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Acute Hepatic Porphyria and Hepatic Porphyrias, run by Alnylam Pharmaceuticals at 12 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 25 Jan 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early (reason given: Sponsor elected not to continue with study). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of participants with current or past clinically symptomatic AHP with urinary aminolevulinic acid (ALA) or porphobilinogen (PBG) values above the upper limit of normal (ULN) (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Jan 2019.